Inherited Retinal Dystrophy Associated With RPE65 Mutations
Investigational drug(s) / intervention(s)
LX101
LX101: Subretinal administration of LX101 to the study eye
Study summary
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
Eligibility
Sex
ALL
Min age
6 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Subject and/or their guardian signing a written informed consent.
Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.
Subjects are 6 years of age or older.
Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.
Exclusion Criteria:
* Prior gene therapy for IRD and other hereditary eye diseases.
Pre-existing eye conditions that would interfere with interpretation of study endpoints.
Active intraocular or periocular infections in the study eye.
Lacking of sufficient surviving retinal cells.
Prior ocular surgery within six months.
Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds.
Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
Primary outcome measure(s)
Mobility Test — 12 months Changes in functional vision from baseline, determined by mobility test score
Trial sites (4)
Facility
City
Region
Status
Zhongshan Ophthalmic Center, Sun Yat sen University
Guangzhou
Guangdong
Southwest Hospital of AMU
Chongqing
China
Shanghai General Hospital
Shanghai
China
Tianjin Medical University Ophthalmology Hospital
Tianjin
China
More Innostellar Biotherapeutics Co.,Ltd trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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